Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site.

ABSTRACT:

Unlabelled

The adeno-associated viruses (AAVs), which are being developed as gene delivery vectors, display differential cell surface glycan binding and subsequent tissue tropisms. For AAV serotype 1 (AAV1), the first viral vector approved as a gene therapy treatment, and its closely related AAV6, sialic acid (SIA) serves as their primary cellular surface receptor. Toward characterizing the SIA binding site(s), the structure of the AAV1-SIA complex was determined by X-ray crystallography to 3.0 Å. Density consistent with SIA was observed in a pocket located at the base of capsid protrusions surrounding icosahedral 3-fold axes. Site-directed mutagenesis substitution of the amino acids forming this pocket with structurally equivalent residues from AAV2, a heparan sulfate binding sero

SUBMITTER: Huang LY 

PROVIDER: S-EPMC4934739 | biostudies-literature | 2016 Jun

REPOSITORIES: biostudies-literature